FDA Tells BrainStorm Evidence Appears Lacking to Support Seeking Approval for ALS Therapy

Rare Daily Staff

The U.S. Food and Drug Administration told BrainStorm Cell Therapeutics in response to a high-level data summary from the company’s ALS phase 3 clinical trial of its autologous stem cell therapy that the current level of clinical data does not provide the threshold of substantial evidence that the agency is seeking to support an application for approval.

The agency, however, advised the company that its recommendation does not preclude Brainstorm from proceeding with an application submission seeking approval for the therapy.

“Brainstorm will first consult with principal investigators, ALS experts, expert statisticians, regulatory advisors, and ALS advocacy groups to assess the benefit/risk of a BLA submission before making a final decision,” said Chaim Lebovits, CEO of Brainstorm Cell Therapeutics.

Amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, is a progressive disease that causes damage to cells in the brain and spinal cord known as motor neurons. Motor neurons transmit signals from the brain to the muscles. When motor neurons become damaged and eventually die, the brain can no longer control muscle actions. The motor neurons affected in ALS are those that initiate and control voluntary movements. With the progressive loss of voluntary muscle action, patients with ALS may lose their ability to speak, eat, move, and breathe.

The NurOwn technology platform is an investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression.

In November, the company reported top line data from a phase 3 study of its NurOwn adult stem cell therapy failed to meet its primary and key secondary endpoints against a placebo.

In a press release announcing the FDA’s recommendation to the company, BrainStorm noted that researchers with experience in ALS therapy development found that there was evidence of benefit from Brainstorm’s NurOwn cell therapy.

“The clear signal in this trial that some patients with ALS respond to treatment with NurOwn is a light at the end of the tunnel,” said Anthony Windebank, professor of neuroscience at Mayo Clinic College of Medicine and Science. “The careful study of biomarkers associated with response will help lead us forward towards a broadly effective therapy.”

Ralph Kern, president and chief medical officer of Brainstorm, said the company will complete all ALS phase 3 study analyses to support scientific communication of the phase 3 data, including a peer-reviewed manuscript.

“The results of the phase 3 clinical trial are very important,” he said, “and we are confident that the analysis of key ALS disease biomarkers will contribute to our understanding of ALS.”

Photo: Chaim Lebovits, CEO Brainstorm Cell Therapeutics